| NAME |
ORIGINATOR |
INDICATION |
DEVELOPMENT STATUS |
| Carbaglu® |
Orphan Europe
(Recordati) |
NAGS deficiency |
Approved in EU
Pre-registration in US |
| Carbaglu® |
Orphan Europe
(Recordati) |
Organic acidaemias |
Pre-registration in EU |
| Vedrop® |
Orphan Europe
(Recordati) |
Vitamin E deficiency in cystic fibrosis
and chronic cholestasis |
Filed with EMEA |
| Infasurf® |
Ony |
Calf derived surfactant for RDS |
Pre-filing |
| Stanate® |
Rockefeller U. / InfaCare |
Neonatal jaundice, hyperbilirubinemia |
Phase II / III |
| Cystagon® |
Mylan |
Other indication unrelated to nephropathic
cystinosis |
Phase II / III |
| Cystadrops® |
Orphan Europe
(Recordati) |
Ocular cystinosis |
Phase II |
| Normosang® |
Orphan Europe
(Recordati) |
Hepatic porphyria |
Approved in EU
Pre-registration in US |
